CA2565103C - Compose oligomere antisens a substitution inosine conjugue a un peptide, et procede associe - Google Patents

Compose oligomere antisens a substitution inosine conjugue a un peptide, et procede associe Download PDF

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CA2565103C
CA2565103C CA2565103A CA2565103A CA2565103C CA 2565103 C CA2565103 C CA 2565103C CA 2565103 A CA2565103 A CA 2565103A CA 2565103 A CA2565103 A CA 2565103A CA 2565103 C CA2565103 C CA 2565103C
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compound
conjugate
string
inosine
bases
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CA2565103A1 (fr
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Patrick L. Iversen
Dwight D. Weller
Jed N. Hassinger
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Sarepta Therapeutics Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A—HUMAN NECESSITIES
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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Abstract

La présente invention se rapporte à un conjugué thérapeutique oligomère-peptide, et à des procédés d'utilisation dudit conjugué. Le conjugué selon l'invention contient : (a) un composé analogue d'oligonucléotide sensiblement non chargé, qui possède une séquence de base renfermant une chaîne de bases qui sont complémentaires à au moins quatre base cytosine contiguës dans une région d'acide nucléique cible à laquelle le composé est destiné à se lier ; et (b) un peptide riche en arginine, conjugué au composé, qui augmente efficacement l'assimilation du composé dans des cellules cibles. La chaîne de bases dans le composés contient au moins une base inosine placée dans la chaîne de façon à limiter le nombre de bases guanine contiguës dans ladite chaîne à trois au plus. Le conjugué selon l'invention présente une assimilation cellulaire supérieure à celle du composé seul, grâce au peptide riche en arginine, et une activité antisens sensiblement supérieure à celle du conjugué en l'absence de substitutions de guanine par inosine.
CA2565103A 2004-05-24 2005-05-24 Compose oligomere antisens a substitution inosine conjugue a un peptide, et procede associe Expired - Lifetime CA2565103C (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US57404804P 2004-05-24 2004-05-24
US60/574,048 2004-05-24
US11/136,245 US20050288246A1 (en) 2004-05-24 2005-05-23 Peptide conjugated, inosine-substituted antisense oligomer compound and method
US11/136,245 2005-05-23
PCT/US2005/018213 WO2005115479A2 (fr) 2004-05-24 2005-05-24 Compose oligomere antisens a substitution inosine conjugue a un peptide, et procede associe

Publications (2)

Publication Number Publication Date
CA2565103A1 CA2565103A1 (fr) 2005-12-08
CA2565103C true CA2565103C (fr) 2014-10-14

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CA2565103A Expired - Lifetime CA2565103C (fr) 2004-05-24 2005-05-24 Compose oligomere antisens a substitution inosine conjugue a un peptide, et procede associe

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US (3) US20050288246A1 (fr)
EP (1) EP1765414B1 (fr)
JP (2) JP4974890B2 (fr)
AT (1) ATE529513T1 (fr)
AU (1) AU2005247460B2 (fr)
CA (1) CA2565103C (fr)
ES (1) ES2375631T3 (fr)
WO (1) WO2005115479A2 (fr)

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ATE529513T1 (de) 2011-11-15
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US8053420B2 (en) 2011-11-08
JP4974890B2 (ja) 2012-07-11
AU2005247460A1 (en) 2005-12-08
WO2005115479A3 (fr) 2007-11-22
EP1765414A2 (fr) 2007-03-28
HK1101314A1 (en) 2007-10-12
AU2005247460B2 (en) 2012-08-02
US20050288246A1 (en) 2005-12-29
CA2565103A1 (fr) 2005-12-08
ES2375631T3 (es) 2012-03-02
EP1765414B1 (fr) 2011-10-19
EP1765414A4 (fr) 2009-10-21
US20080182973A1 (en) 2008-07-31
US8877725B2 (en) 2014-11-04
WO2005115479A2 (fr) 2005-12-08

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